FDA Approves Lirafugratinib After 46% Response in Bile Duct Cancer

The FDA approved lirafugratinib (Lyrfigtu, Elevar Therapeutics), an oral kinase inhibitor, for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma harboring a fibroblast growth factor receptor 2 (FGFR2) gene fusion or other rearrangement. The agency announced the approval on September 23, 2026.

The recommended dose is 70mg orally once daily until disease progression or unacceptable toxicity. The application received priority review, and lirafugratinib had received breakthrough therapy and orphan drug designations. Elevar Therapeutics said it expects US availability by the fourth quarter of 2026.

Efficacy was evaluated in REFOCUS, a multicenter, open-label, single-arm trial involving 116 patients with unresectable or metastatic cholangiocarcinoma who had received chemotherapy or chemoimmunotherapy but no prior FGFR inhibitor. Patients received lirafugratinib 70mg daily. The principal efficacy measures were objective response rate and duration of response, assessed by an independent review committee using RECIST version 1.1.

The objective response rate was 46% (95% CI, 36%-55%), and median duration of response was 11.8 months (95% CI, 7.5-13.0 months). Elevar also reported a median progression-free survival of 11.3 months. REFOCUS was a single-arm trial without a comparator group.

The FDA identified ocular toxicity, hyperphosphatemia and soft tissue mineralization, and embryo-fetal toxicity among the prescribing information's warnings and precautions. In safety data reported by Elevar from 385 lirafugratinib recipients, retinal pigment epithelial detachment occurred in 31%, including grade 3 events in 1.8%. Serious adverse reactions occurred in 32% of patients in the company's safety summary.

Sources: US Food and Drug Administration. FDA approves lirafugratinib for previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma. US Food and Drug Administration; September 23, 2026. Accessed September 24, 2026.

Elevar Therapeutics. Elevar Therapeutics announces FDA approval of Lyrfigtu (lirafugratinib) as second-line cholangiocarcinoma with FGFR2 fusion or other rearrangement treatment option. Press release. Elevar Therapeutics; September 23, 2026. Accessed September 24, 2026.